RNA Medicines

Arcturus is developing RNA medicines to treat the root-cause of disease and help patients in a truly innovative way. Our current focus is on RNA medicines for the treatment of rare diseases and high unmet medical needs. Arcturus has developed a novel, potent and safe RNA Therapeutics platform called LUNAR™, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, antisense and microRNA oligotherapeutics. In addition, we incorporate Unlocked Nucleic Acid (UNA) chemistry into the oligonucleotide drug candidate enabling the targeting of any gene in the human genome.  Our lead asset is an UNA-modified, LUNAR-formulated siRNA targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis. The goals of our development strategy are to demonstrate a more favorable dosing regimen, superior potency and safety profile — to clinically differentiate our candidate versus other TTR drug candidates.

Name Indication API Discovery Research Preclinical Clinical
Liver
LUNAR-101 Cardiomyopathy UNA
LUNAR-102 Polyneuropathy UNA
Name Indication API Discovery Research Preclinical Clinical
Partnered
Tekmira Hepatitis B Virus UNA
© 2014 Arcturus Therapeutics, Inc.